We received numerous calls and emails concerning Biogen-Idec’s recent announcement of clinical trial results demonstrating that the experimental drug, dexpramipexole, did not significantly alter disease progression or extend the life of patients with ALS. These results are, indeed, disappointing to all patients, friends and family directly affected by this devastating disease as well as those involved in the care and treatment of ALS patients. Despite years of research, millions of dollars spent and tremendous efforts by many to make new treatment options available, ALS remains a disease that claims too many lives each year.

In this letter I hope to clarify the difference of therapeutic approach between dexpramipexole and our ALS program.

Biogen-Idec licensed dexpramipexole from Knopp Biosciences in 2010 for development as a new treatment for ALS. It is a synthetic chemical, small molecule drug that has a number of significant differences from the therapeutic approach represented by our collaboration with Biogen-Idec. Dexpramipexole is a molecule that is thought to enhance nerve cell mitochondrial function (the internal power source for cellular life and activity) and protect neurons under stressors like those induced by ALS. This drug is required to penetrate neurons and the mitochondria in order to have this therapeutic effect.